Peshkov
To kick things off ROME Therapeutics based in Cambridge, MA secured $77 million from new and existing investors in a series B round. ROME is developing novel therapies for cancer and autoimmune diseases by harnessing the power of the repeatome–long stretches of unmapped repetitive sequences of nucleic acids long dismissed as ‘junk DNA’. Having identified several repeatome-derived drug targets, and with multiple discovery programs underway, the funding will be used to advance the company’s lead programs into the clinic and expand its repeatomics platform.
Clinical-stage gene therapy company LEXEO Therapeutics located in New York, NY closed a sizeable $100 million series B financing from new and existing investors. LEXEO is advancing adeno-associated virus-mediated disease-modifying treatments for devastating genetic CNS and cardiovascular conditions, including Alzheimer’s disease, Batten disease, and cardiomyopathy associated with Friedreich’s ataxia.
Meanwhile, Skyhawk Therapeutics raised $133 million in its latest round, bringing total equity funding plus partnership capital in the company to over $600 million to date. Addressing the underlying gene expression of disease, Skyhawk is developing small molecule therapeutics that correct RNA expression. The company’s pipeline programs, many of which are against targets formerly considered ‘undruggable’ for traditional protein-targeting mechanisms, include cancer, neurodegenerative, autoimmune, inflammatory, and infectious diseases. The investment will allow Skyhawk to accelerate development of its novel small molecule RNA-modifying drug candidates into the clinic.
Aiming to revolutionize genetic medicine manufacturing, Touchlight Genetics based in London, UK extended its most recent private investment round to $125 million. Instead of manufacturing plasmid DNA using restrictive bacterial fermentation, Touchlight has developed a novel, synthetic DNA vector, known as ‘doggybone’ or dbDNA, and an in vitro enzymatic manufacturing process using benchtop equipment that can be more-easily scaled and eliminates the need for bacterial sequences, including antibiotic resistance genes. Consequently, Touchlight can produce DNA at unprecedented speed (weeks versus months), scale and purity. The funding will be used to increase manufacturing capacity and advance the company’s pipeline, which includes DNA vaccines for both Covid-19 and head and neck cancer.
October financings
Private biopharma companies closing financing rounds in October included two focussed on regulatory T cells (Tregs) immunotherapies. Emerging biotech Egle Therapeutics located in Paris, France, spun out of Institut Curie, is developing first-in-class immunotherapies targeting immune suppressor Tregs for oncology and autoimmune diseases. The company has a unique approach of ‘Tregs starving’ to specifically target the most immunosuppressive Treg targets with computationally designed immunocytokines acting as antagonists or selective agonists. Egle raised €40 million ($46.4 million) in a series A financing from an international investor syndicate, including Takeda Ventures. The new capital will be used primarily to advance two lead Treg-targeting immunocytokines into the clinic and further strengthen its internal drug pipeline.
Another Treg company, Mozart Therapeutics based in Seattle, WA, launched with a $55 million series A financing. In a novel approach to tackling a root cause of immune system dysregulation, Mozart is targeting and modulating a CD8 (not the typical CD4) Treg network that plays a key role in ‘managing’ autoreactive and pathogenic immune cells that can cause autoimmune and inflammatory disease. The company’s CD8 Treg modulators selectively restore the intrinsic functions of CD8 Tregs to counteract early events in the autoimmune inflammatory cascade, resetting and restoring the immune system balance. The funds will be used to progress Mozart’s first-in-class disease-modifying therapeutic candidate against coeliac disease, and other pipeline programs.
Meanwhile, DNA synthesis and South San Francisco-based company DNA Script raised $165 million in an oversubscribed series C financing from new and existing investors. DNA Script is pioneering enzymatic DNA synthesis and DNA printing on demand; the company’s fully automated instrument enables in-house custom DNA printing (up to 96 oligos within hours) without using toxic organic chemicals or extended delivery wait times from third-party DNA service providers. The new funds, which bring the total raised by the company to $280 million, will help commercialize its SYNTAX, the world’s first benchtop DNA printer powered by enzymatic technology.
Also closing an oversubscribed series C financing, Cambrian Biopharma based in New York, NY, raised $100 million from new and existing investors to advance health span-boosting therapeutics to the clinic. Cambrian operates as a distributed development company (DisCo) model that the company says combines the advantages of a venture capital firm and a big pharmaceutical company with the nimbleness of a biotech startup. Cambrian currently has 14 novel therapeutics in development across its majority-held pipeline companies, each therapeutic targeting a different biological driver of aging. The funding, which brings the total raised to approximately $160 million since the company was founded in 2019, will be used to expand and progress the pipeline, including the start of clinical trials for three programs in 2023.
November financings
Arbor Biotechnologies based in Cambridge, MA closed a $215 million oversubscribed series B financing to advance next-generation precision editing therapeutics. Arbor’s discovery engine applies machine learning and AI to mine its database containing billions of proteins. The result, says the company, is the most extensive toolbox of wholly owned CRISPR genomic editors in the industry. Arbor and its partners can tailor these to edit or rewrite the genetic errors that result in numerous disease pathologies, resulting in potentially curative medicines. The proceeds, which bring the amount raised to over $300 million since the company’s founding in 2016, will be used in part to advance its lead programs in liver and CNS disease into the clinic and progress a pipeline of precision editing therapeutics.
Also based in Cambridge, MA is epigenetic editing start-up Chroma Medicine that launched with $125 million in seed and series A financing. Chroma’s modular epigenetic editors can be precisely programmed to durably silence, activate or alter the expression of several genes at once. Although it’s early days, altering gene expression should be more precise and versatile than fundamentally changing or cutting the underlying DNA sequence, with the potential to address complex diseases and with fewer unanticipated side effects.
Meanwhile, bit.bio located in Cambridge, UK and San Francisco, CA raised $103 million in the first close of a series B financing from new and existing institutional and strategic investors. Combining synthetic and stem-cell biology, the company’s cell coding technology, opti-ox, reliably activates specific transcription factors within the cells. This enables unlimited batches of any human cell to be manufactured consistently at scale through direct reprogramming of stem cells. bit.bio has already launched two products for research and drug discovery—glutamatergic neurons and skeletal myocytes—and the new funding will support continued growth of its portfolio of unique physiologically-relevant human cell models.
Quell Therapeutics (London, UK and Boston, MA, USA) raised $156 million in an oversubscribed series B financing to advance its pioneering (autologous) multi-modular engineered Treg cell therapy pipeline and platform. By harnessing Tregs, novel cell therapies can be designed to suppress overactive immune responses, drive long-term tolerance in the local immune environment and promote tissue repair. Quell says it can design, engineer (by knocking in additional genes) and manufacture at scale, therapeutic Treg products with greater stability, persistence and potency than earlier generations. The financing will be used in part to further clinical development of QEL-001, a first-in-class antigen-specific multi-modular CAR-Treg cell therapy candidate designed to prevent transplanted liver rejection.
Also in November, clinical-stage biopharma company ACELYRIN Inc located in Los Angeles, CA closed a huge $250 million series B financing and in-licensed Izokibep (from Affibody AB), a unique late-stage antibody mimetic, interleukin-17A inhibitor to treat several autoimmune/inflammatory diseases. Izokibep, which is about a tenth the size of a monoclonal antibody, has been designed for enhanced tissue penetration and high exposures in a single subcutaneous injection—overcoming the limitations of monoclonal antibodies. The series B proceeds will be used to accelerate izokibep development, and fund licensing and acquisition of additional immunology programs.
Finally Antios Therapeutics based in Mendham, NJ, a clinical-stage biopharmaceutical company focused on viral diseases, raised $96 million in a series B financing, which will support the ongoing phase 2 program of lead candidate ATI-2173 in hepatitis B. ATI-2173 is a novel, orally administered, liver-targeted active site polymerase inhibitor nucleotide (ASPIN) that can shut down hepatitis B viral polymerase activity and viral replication. ATI-2173 has the potential to become an integral part of a curative combination regimen for chronic hepatitis B.
